AeroVanc in the Treatment of Methicillin-resistant Staphylococcus Aureus Infection in Patients With Cystic Fibrosis
- Conditions
- Cystic FibrosisMRSA
- Interventions
- Drug: Placebo inhalation powder
- Registration Number
- NCT03181932
- Lead Sponsor
- Savara Inc.
- Brief Summary
This is a multi-center, randomized phase III study to evaluate the clinical effectiveness of AeroVanc in persistent methicillin-resistant Staphylococcus aureus (MRSA) infection in patients with cystic fibrosis (CF).
- Detailed Description
This is a phase III, randomized, multicenter, double-blind, placebo-controlled, parallel-group study to examine the safety and efficacy of AeroVanc in the treatment of persistent MRSA lung infection in patients diagnosed with CF. After the Screening period to confirm study eligibility, participants are randomly assigned in a blinded fashion to receive either AeroVanc 30 mg twice daily (BID), or placebo BID (1:1 active to placebo) by inhalation for 24 weeks or 3 dosing cycles (Period 1). Upon completion of Period 1, participants receive open-label AeroVanc 30 mg BID for an additional 24 weeks or 3 dosing cycles (Period 2), to evaluate long-term safety of AeroVanc. A dosing cycle is defined as 28 days of treatment followed by 28 days of observation.
Participants on a 28-day cyclical on/off anti-Pseudomonal antibiotic regimen enter the Screening period at a time such that the Baseline visit coincide with the end of their anti-Pseudomonas antibiotic cycle. Study drug is thereby administered during the off-cycle, and participants can then resume anti-Pseudomonal therapy during the 28-day observation period. Participants continuing alternating anti-Pseudomonal therapy can continue their treatment during the study drug administration, and observation period.
The primary and secondary analyses are conducted in participants ≤21 years old. Subjects \>21 years old are analyzed separately as supportive analyses.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 188
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Open-label vancomycin inhalation powder Vancomycin inhalation powder In the 24-week Period 2, all participants receive AeroVanc 30 mg BID by inhalation during three dosing cycles, each cycle being 28 days of treatment followed by 28 days of observation. Double-blind vancomycin inhalation powder Vancomycin inhalation powder Vancomycin inhalation powder 30 mg is administered twice daily (BID) during the 24-week double-blind period (Period 1) by inhalation during three dosing cycles, each cycle being 28 days of treatment followed by 28 days of observation. Double-blind placebo inhalation powder Placebo inhalation powder Matching placebo is administered BID during the 24-week double-blind period (Period 1) by inhalation during three dosing cycles, each cycle being 28 days of treatment followed by 28 days of observation.
- Primary Outcome Measures
Name Time Method Absolute Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Percent Predicted Baseline and Week 4, 12 and 20 The mean absolute change from baseline in FEV1 percent predicted was analyzed sequentially at Week 4 (end of Cycle 1), Week 12 (end of Cycle 2) and at Week 20 (end of Cycle 3).
- Secondary Outcome Measures
Name Time Method Time to First Pulmonary Exacerbation Week 20 Time to first pulmonary exacerbation requiring use of another antibiotic medication (oral, IV, and/or inhaled). The Outcome Measure Data presented are the median percentiles and 95% confidence intervals from Kaplan-Meier estimates.
Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Scores Baseline and Week 4, 12, and 20 The CFQ-R was administered every two weeks using a hand-held e-Diary. CFQ-R scores range between 0 and 100, where higher scores indicate a better outcome. The CFQ-R measures functioning in a variety of domains, including Physical Functioning, Vitality, Health Perceptions, Respiratory Symptoms, Treatment Burden, Role Functioning, Emotional Functioning and Social Functioning. The Outcome Measure Data presented are the Respiratory Symptoms Scores.
Number of Successful Response Cycles Week 20 The number of successful response cycles a participant achieves over Period 1. A response in a cycle is defined by at least a 5 % relative improvement in FEV1 percent predicted at the end of each the respective cycle.
Area Under the FEV1-time Profile Week 20 The mean treatment difference in FEV1 across all post-baseline visits
Frequency of Pulmonary Exacerbations Week 20 The number of pulmonary exacerbations during Period 1 adjusted for the length of follow-up.
Change From Baseline in Cystic Fibrosis Respiratory Symptom Diary-Chronic Respiratory Symptom Score (CFRSD-CRISS) Scores Baseline and Week 4, 12 and 20 The CFRSD-CRISS was administered every two weeks using a hand-held e-Diary. Scores range between 0 and 100, where higher scores indicate a worse outcome.
Relative Change in FEV1 Percent Predicted Baseline and Week 4, 12 and 20 The mean relative change from Baseline in FEV1 percent predicted
Trial Locations
- Locations (71)
Pulmonary Associates of Mobile
🇺🇸Mobile, Alabama, United States
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
University of Arkansas for Medical Sciences
🇺🇸Little Rock, Arkansas, United States
Miller Childrens Hospital MemorialCare Health System Pediatric Pulmonology
🇺🇸Long Beach, California, United States
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
University of Southern California Keck Medical Center of USC
🇺🇸Los Angeles, California, United States
UC Davis Medical Center
🇺🇸Sacramento, California, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
National Jewish Health Adult Cystic Fibrosis Center
🇺🇸Denver, Colorado, United States
Children's National Medical Center
🇺🇸Washington, District of Columbia, United States
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